Ignite Creation Date:
2025-12-24 @ 11:40 PM
Ignite Modification Date:
2026-02-11 @ 3:21 PM
Study NCT ID:
NCT02097251
Status:
NO_LONGER_AVAILABLE
Last Update Posted:
2017-02-06
First Post:
2014-03-24
Is Gene Therapy:
True
Has Adverse Events:
False
Brief Title:
An Open-Label Treatment Protocol With UX003 rhGUS Enzyme Replacement Therapy for an Advanced Stage MPS 7 Patient
Sponsor:
Joyce Fox
Organization:
Study Overview
Official Title:
An Open-Label Treatment Protocol With UX003 Recombinant Human Beta-glucuronidase (rhGUS) Enzyme Replacement Therapy for an Advanced Stage Mucopolysaccharidosis Type 7 (MPS 7) Patient
Status:
NO_LONGER_AVAILABLE
Status Verified Date:
2017-02
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
No
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
None
Brief Summary:
Emergency access granted to treat a single patient with advanced Mucopolysaccharidosis Type 7 with UX003 Recombinant Human Beta-glucuronidase (rhGUS) Enzyme Replacement Therapy via IV administration every other week (QOW) for up to 144 weeks.
Detailed Description:
None
Study Oversight
Has Oversight DMC:
Is a FDA Regulated Drug?:
Is a FDA Regulated Device?:
Is an Unapproved Device?:
Is a PPSD?:
Is a US Export?:
Is an FDA AA801 Violation?: